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. Author manuscript; available in PMC: 2023 Jul 1.
Published in final edited form as: Nat Genet. 2022 Dec 15;55(1):34–43. doi: 10.1038/s41588-022-01257-y

Extended Data Figure 4. Candidate transcript off-targets introduced by common genetic variants for non-CRISPR sequence-based RNA-targeting therapeutic strategies.

Extended Data Figure 4.

a) A common SNP (in blue) introduces a candidate CDS off-target site with 2 mismatches for the FDA-approved antisense oligo Nusinersen. b) Top 1000 candidate transcript off-targets ranked by mismatches and bulges for Nusinersen from a search performed with the 1000G and HGDP genetic variant datasets. c) A common insertion variant (in red) introduces a candidate 3’UTR off-target site with 4 mismatches + bulges for the FDA-approved RNAi therapy Inclisiran. d) Top 1000 candidate transcript off-targets ranked by mismatches and bulges for Inclisiran from a search performed with the 1000G and HGDP genetic variant datasets.